Division of Hematologic Malignancies and Cellular Therapeutics, Department of Internal Medicine, The University of Kansas Medical Center, Kansas City, Kansas, USA.
Department of Pathology and Laboratory Medicine, The University of Kansas Medical Center, Kansas City, Kansas, USA.
J Thromb Haemost. 2022 Oct;20(10):2197-2203. doi: 10.1111/jth.15822. Epub 2022 Aug 3.
ADAMTS13, a plasma metalloprotease that cleaves von Willebrand factor, is crucial for normal hemostasis. Acquired autoantibody-mediated deficiency of plasma ADAMTS13 results in a potentially fatal blood disorder, immune thrombotic thrombocytopenic purpura (iTTP). Plasma ADAMTS13 protease appears to exist in multiple conformations. Under physiological conditions, plasma ADAMTS13 exists predominantly in its "closed" conformation (or latent form), which may be activated by lowering pH, ligand binding, and binding of an antibody against the distal domains of ADAMTS13. In patients with iTTP, polyclonal antibodies target at various domains of ADAMTS13. However, nearly all inhibitory antibodies bind the spacer domain, whereas antibodies that bind the distal C-terminal domains may activate ADAMTS13 through removing its allosteric inhibition. Additionally, the anti-C-terminal antibodies may alter the potency of inhibitory antibodies towards ADAMTS13 activity. This review summarizes some of the most recent knowledge about the ADAMTS13 conformation and its mechanism of inhibition by its autoantibodies.
ADAMTS13 是一种血浆金属蛋白酶,可切割血管性血友病因子,对正常止血至关重要。获得性自身抗体介导的血浆 ADAMTS13 缺乏会导致潜在致命的血液疾病,即免疫性血栓性血小板减少性紫癜(iTTP)。血浆 ADAMTS13 蛋白酶似乎存在多种构象。在生理条件下,血浆 ADAMTS13 主要以“封闭”构象(或潜伏形式)存在,这种构象可通过降低 pH 值、配体结合以及与 ADAMTS13 远端结构域的抗体结合而被激活。在 iTTP 患者中,多克隆抗体针对 ADAMTS13 的不同结构域。然而,几乎所有的抑制性抗体都结合间隔区,而结合远端 C 末端结构域的抗体可能通过去除其变构抑制来激活 ADAMTS13。此外,抗 C 末端抗体可能改变针对 ADAMTS13 活性的抑制性抗体的效力。本综述总结了 ADAMTS13 构象及其自身抗体抑制机制的一些最新知识。